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The four projects in this Program Project share the common
theme of characterizing and understanding normal and abnormal
patterns of gene expression in developing muscle from the
stem cell stage through to maturity.
Project 1: The
Kunkel Laboratory
Gene expression and biochemical studies of filamin/sarcoglycan-related
dystrophies.
Project 2: The
Brown Laboratory (Day Neuromuscular Research Laboratory)
Gene expression and biochemical studies of dysferlin-related
dystrophies.
Project 3: The
Beggs Laboratory
Gene Expression and Biochemical Analysis of Muscle Development
in Myotubular Myopathy.
Project 4: The
Gussoni Laboratory
Gene expression in, and therapeutic application of,
muscle stem cells.
The Program PIs have a long and extensive history of
interaction and have exchanged many ideas, biopsy samples,
and other reagents over many years, and this research project
is designed to further strengthen these collaborations and
to produce synergistic results that are beyond the scope of
any one laboratory. The services of the Clinical Specimen
and Data Core (Core B) will be an important means by which
the Harvard Neuromuscular Disease Project will meet these
goals. The techniques required to perform the aims of this
Core are standard within the laboratories of each PI. By centralizing
these techniques, formalizing data and tissue collections,
and developing a database within the Muscular Dystrophy Research
Portal (MDRP), Core B will increase efficiency and provide
standardization to the analysis of data for each Project and
the Program overall.
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